Related Experiment Video
Updated: May 26, 2025
09:51
Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
Published on: May 25, 2018
33.7K
Advanced delivery systems for gene editing: A comprehensive review from the GenE-HumDi COST Action Working Group
Alessia Cavazza1,2, Francisco J Molina-Estévez3,4,5, Álvaro Plaza Reyes6
1Molecular and Cellular Immunology Section, Department of Infection, Immunity & Inflammation, UCL Great Ormond Street Institute of Child Health, University College London, 20 Guilford Street, London WC1N 1DZ, UK.
Molecular Therapy. Nucleic Acids
|February 24, 2025
Summary
Genome editing offers precise genetic modification for therapies. This review details viral and non-viral delivery systems for genome editing, crucial for clinical applications.
Area of Science:
- Biotechnology
- Molecular Biology
- Gene Therapy
Background:
- Genome editing technologies, utilizing programmable DNA nucleases, enable precise genetic modifications.
- These technologies hold promise as alternatives to traditional therapeutic strategies.
- Clinical translation of genome editing is challenged by efficient and safe delivery of editing reagents.
Purpose of the Study:
- To review and summarize the diverse delivery systems for genome editing components.
- To highlight the advantages and limitations of various delivery platforms.
- To discuss the potential clinical applications of these delivery systems.
Main Methods:
- Comprehensive literature review of viral and non-viral delivery systems for genome editing.
- Analysis of system efficiency, safety, and scalability.
- Discussion of current challenges and future directions in the field.
Main Results:
- A wide array of delivery systems, including viral vectors (e.g., AAV, lentivirus) and non-viral methods (e.g., lipid nanoparticles, electroporation), are available.
- Each system presents unique advantages and limitations regarding cargo capacity, immunogenicity, targeting efficiency, and manufacturing.
- The choice of delivery system significantly impacts the feasibility and success of genome editing interventions.
Conclusions:
- Effective delivery of genome editing tools is paramount for successful clinical translation.
- Ongoing advancements in both viral and non-viral delivery systems are expanding therapeutic possibilities.
- Further research and development are needed to optimize delivery strategies for enhanced safety and efficacy in clinical settings.
Keywords:
COSTEuropean Cooperation in Science and TechnologyGenE-HumDiMT: Delivery Strategiesbase editorsdelivery systemsgenome editingregulatory guidelinesMore Related Videos
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